Congenital adrenal hyperplasia due to 21-hydroxylase deficiency

Neurocrine Biosciences Presents Post Hoc Data Analysis in Congenital Adrenal Hyperplasia at ESPE 2023

Retrieved on: 
Thursday, September 21, 2023
CAH, Neurocrine Biosciences, ESPE, Antidepressant discontinuation syndrome, Adrenocorticotropic hormone, Congenital adrenal hyperplasia due to 21-hydroxylase deficiency, Vital signs, Patient, SAN, Adolescence, Androstenedione, European Society for Phenylketonuria and Allied Disorders Treated as Phenylketonuria, ACTH, GC, Safety, Pharmaceutical industry, Birth control, The Hague, A4

SAN DIEGO, Sept. 21, 2023 /PRNewswire/ -- Neurocrine Biosciences, Inc. (Nasdaq: NBIX), today announced that it will present a new post hoc analysis of Phase 2 data of the investigational drug crinecerfont in adolescent patients with classic congenital adrenal hyperplasia (CAH) due to 21-hydroxylase deficiency (21-OHD).

Key Points: 
  • SAN DIEGO, Sept. 21, 2023 /PRNewswire/ -- Neurocrine Biosciences, Inc. (Nasdaq: NBIX), today announced that it will present a new post hoc analysis of Phase 2 data of the investigational drug crinecerfont in adolescent patients with classic congenital adrenal hyperplasia (CAH) due to 21-hydroxylase deficiency (21-OHD).
  • These new data will be presented at the 61st Annual European Society for Pediatric Endocrinology ( ESPE ) Meeting in The Hague, Netherlands from Sept. 21–23, 2023.
  • Post hoc analysis of these data released today assessed whether baseline hormone concentration and glucocorticoid (GC) doses correlated with response to treatment.
  • We plan to announce top-line data from our Phase 3 CAHtalyst™ Pediatric Study in early Q4 2023."

Neurocrine Biosciences Presents Data on Treatment of Patients with Congenital Adrenal Hyperplasia at ECE 2023

Retrieved on: 
Friday, May 12, 2023
EEA, CRF1, Androgen, Congenital adrenal hyperplasia, Civil service commission, Virilization, CAH, Enzyme, Sleep, Neurocrine Biosciences, Neurocrine, Infertility, Hydrocortisone, Appetite, Adrenocorticotropic hormone, Gross metropolitan product, Congenital adrenal hyperplasia due to 21-hydroxylase deficiency, Congress, Insomnia, Bruise, Disorder, Dizziness, Poster, Patient, Addison's disease, Physician, SAN, European Economic Area, Common, European, European Society of Endocrinology, Public, Nausea, Androstenedione, Growth, Medicines and Healthcare products Regulatory Agency, ECE, Upper respiratory tract infection, ACTH, GC, Headache, Diurnal, European Commission, MHRA, Fatigue, Pharmaceutical industry, Birth control, Endocrinology, Fever

Post hoc analyses of these data released today assessed whether baseline hormone levels and glucocorticoid (GC) doses correlated with treatment response.

Key Points: 
  • Post hoc analyses of these data released today assessed whether baseline hormone levels and glucocorticoid (GC) doses correlated with treatment response.
  • See the abstract (Poster #274; Response to Crinecerfont Treatment in Adults with Classic Congenital Adrenal Hyperplasia is Correlated with Elevated Baseline Hormone Levels, but Not Glucocorticoid Dose) for more information here .
  • "Androgen excess is a hallmark feature of classic congenital adrenal hyperplasia.
  • These data will be part of an oral presentation (Oral Presentation, May 15, 2:20–2:30pm: Switching Patients with Congenital Adrenal Hyperplasia to Modified-Release Hydrocortisone Capsules: Relative Bioavailability and Disease Control).

Global Congenital Adrenal Hyperplasia Market Report to 2032 - Insights, Epidemiology and Forecasts - ResearchAndMarkets.com

Retrieved on: 
Friday, October 14, 2022
Biotechnology, Pharmaceutical, Health, Androgen, Disease, 21-Hydroxylase, Review, Diagnosis, Infant, Congenital adrenal hyperplasia, Patient, CAH, Research, Enzyme, Adrenal crisis, Genetic distance, Aldosterone, Kidney, Congenital adrenal hyperplasia due to 21-hydroxylase deficiency, Mineralocorticoid, Awareness, Birth defect, Hormone, Death, Exocrine gland, Algorithm, Pharmaceutical industry

It covers the details of conventional and current medical therapies and diagnoses available in the congenital adrenal hyperplasia (CAH) market to treat the condition.

Key Points: 
  • It covers the details of conventional and current medical therapies and diagnoses available in the congenital adrenal hyperplasia (CAH) market to treat the condition.
  • The congenital adrenal hyperplasia (CAH) Market Report gives a thorough understanding of congenital adrenal hyperplasia (CAH) by including details of disease definition, symptoms, causes, pathophysiology, and diagnosis.
  • It also provides the treatment algorithms and treatment guidelines for congenital adrenal hyperplasia (CAH) in the US, Europe, and Japan.
  • The launch of emerging therapies will significantly impact the congenital adrenal hyperplasia (CAH) market.

BridgeBio Pharma Announces Dosing of First Patient in Phase 1/2 Trial of Investigational Gene Therapy for Congenital Adrenal Hyperplasia (CAH)

Retrieved on: 
Thursday, January 27, 2022
LinkedIn, Heart, Securities Exchange Act of 1934, SEC, AAV, Industry, Lists of diseases, Cancer, Annual report, CAH, Exocrine gland, Patient, Adventure, PALO, Congenital adrenal hyperplasia, GLOBE, A4, Growth, Nasdaq, Clinical trial, Hormone, U.S. Securities and Exchange Commission, University, Congenital adrenal hyperplasia due to 21-hydroxylase deficiency, Time, Science, TMC1, Biomarker, Safety, European Medicines Agency, Research, Aldosterone, Canavan disease, Center, Twitter, Securities Act of 1933, Disease, Achievement, Environment, Tuberous sclerosis, Human, CEO, FDA, Food, Risk, COVID-19, Hyperplasia, Drug Quality and Security Act, Hope, Therapy, Androstenedione, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Mutation, Cystinuria, Charalambos Sarafoglou, Vaccine, Pharmaceutical industry

PALO ALTO, Calif., Jan. 27, 2022 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (Nasdaq: BBIO) (“BridgeBio” or the “Company”), a commercial-stage biopharmaceutical company focused on genetic diseases and cancers, today announced that the first patient has been dosed in ADventure, its Phase 1/2 clinical trial of BBP-631, an investigational adeno-associated virus (AAV) 5 gene therapy for the treatment of classic congenital adrenal hyperplasia (CAH). CAH is one of the most prevalent genetic diseases, with more than 75,000 cases estimated in the United States and European Union.

Key Points: 
  • Our investigational gene therapy offers patients a potential single-dose intervention designed to restore their bodys hormone and steroid balance by making their own cortisol and aldosterone, said Eric David, M.D., J.D., CEO at BridgeBio Gene Therapy.
  • This is the second gene therapy trial we have initiated in less than four months, and we are excited to advance this trial and our other gene therapy programs in the hope of improving patients lives.
  • As an endocrinologist, its incredibly exciting to reimagine a new approach to treating this disease, added Adam Shaywitz, M.D., Ph.D., chief medical officer at BridgeBio Gene Therapy.
  • Adrenas Therapeutics, the affiliate company of BridgeBio focused on developing BBP-631 for CAH, is part of BridgeBio Gene Therapys portfolio.

Spruce Biosciences Presents Phase 1 and 2 Data for Tildacerfont in Adults with Congenital Adrenal Hyperplasia from Endocrine Society’s 2021 Annual Meeting

Retrieved on: 
Wednesday, March 17, 2021
Biotechnology, Pharmaceutical, Health, Clinical trials, Organ systems, Medical specialties, Medicine, Adrenal gland disorders, Congenital adrenal hyperplasia, 17α-Hydroxyprogesterone, Hyperplasia, Adrenal gland, CAH, Congenital adrenal hyperplasia due to 21-hydroxylase deficiency, Congenital adrenal hyperplasia due to 11β-hydroxylase deficiency, Tildacerfont, Spruce Biosciences

Spruce Biosciences, Inc. (Nasdaq: SPRB), a late-stage biopharmaceutical company focused on developing and commercializing novel therapies for rare endocrine disorders with significant unmet need, presented data from its Phase 1 and 2 programs of tildacerfont in adults with classic congenital adrenal hyperplasia (CAH) from the Endocrine Societys 2021 Annual Meeting , taking place virtually March 20 23, 2021.

Key Points: 
  • Spruce Biosciences, Inc. (Nasdaq: SPRB), a late-stage biopharmaceutical company focused on developing and commercializing novel therapies for rare endocrine disorders with significant unmet need, presented data from its Phase 1 and 2 programs of tildacerfont in adults with classic congenital adrenal hyperplasia (CAH) from the Endocrine Societys 2021 Annual Meeting , taking place virtually March 20 23, 2021.
  • Participants in the study underwent concurrent salivary and serum concentration measurements of androstenedione (A4), 17-hydroxyprogesterone (17-OHP) and testosterone (T).
  • Spruce Biosciences is a late-stage biopharmaceutical company focused on developing and commercializing novel therapies for rare endocrine disorders with significant unmet need.
  • Spruce is initially developing its wholly-owned product candidate, tildacerfont, as the potential first non-steroidal therapy for patients suffering from classic congenital adrenal hyperplasia (CAH).

Neurocrine Biosciences Reports Positive Phase II Data for Crinecerfont in Adults with Congenital Adrenal Hyperplasia at ENDO Online 2020

Retrieved on: 
Monday, June 8, 2020
Adrenal gland disorders, Endocrine diseases, Medicine, Branches of biology, Congenital adrenal hyperplasia due to 21-hydroxylase deficiency, 21-Hydroxylase, Glucocorticoid, Adrenal gland, Congenital adrenal hyperplasia, Lipoid congenital adrenal hyperplasia

These data suggest that crinecerfont has the potential to improve CAH symptoms and to reduce the burden of daily glucocorticoid exposure for these patients.

Key Points: 
  • These data suggest that crinecerfont has the potential to improve CAH symptoms and to reduce the burden of daily glucocorticoid exposure for these patients.
  • Neurocrine Biosciences plans to initiate a single, global registrational study of crinecerfont in adult patients with classic CAH in the second half of 2020.
  • Classic CAH is a genetic disorder, in which an enzyme deficiency alters the production of adrenal steroids.
  • The Phase II open-label, multiple-dose, dose-finding study assessed the safety, tolerability, pharmacokinetics and pharmacodynamics of crinecerfont in 18 adults with classic 21-hydroxylase deficiency CAH.

Spruce Biosciences Announces Launch of CAHstudy.com to Initiate Screening of Patients for CAH Clinical Program

Retrieved on: 
Tuesday, May 12, 2020
Biotechnology, Pharmaceutical, Health, Clinical trials, Adrenal gland disorders, Medicine, Rare diseases, Health, Medical specialties, Epidemiology, Neonatology, Newborn screening, CAH, Congenital adrenal hyperplasia due to 21-hydroxylase deficiency, Congenital adrenal hyperplasia, Congenital Adrenal Hyperplasia (CAH), Tildacerfont, Spruce Biosciences

In September 2019, Spruce Biosciences announced positive results from a Phase 2, multicenter, 12-week study of tildacerfont in adult patients with Classic CAH.

Key Points: 
  • In September 2019, Spruce Biosciences announced positive results from a Phase 2, multicenter, 12-week study of tildacerfont in adult patients with Classic CAH.
  • Our previous studies have demonstrated promising safety and efficacy results from the use of tildacerfont in adults with Classic CAH, said Richard King, Chief Executive Officer, Spruce Biosciences.
  • We are excited to move forward with the CAHmelia clinical program and encourage patients with CAH and their families to visit CAHstudy.com to register their interest in the study and begin the screening process.
  • Although CAH testing is part of the newborn screening program, the only approved therapy for CAH are glucocorticoids.

Millendo Therapeutics Announces 21st European Congress of Endocrinology 2019 Presentations on Livoletide for Prader-Willi Syndrome and Nevanimibe for Classic Congenital Adrenal Hyperplasia

Retrieved on: 
Wednesday, May 8, 2019
Health, Biotechnology, Clinical trials, Pharmaceutical, Endocrine diseases, Adrenal gland disorders, Organ systems, Endocrinology, Adrenal insufficiency, Congenital adrenal hyperplasia, Adrenal gland, Cushing's syndrome, Hyperplasia, CAH, Lipoid congenital adrenal hyperplasia, Congenital adrenal hyperplasia due to 21-hydroxylase deficiency

Livoletide, a potential first-in-class treatment for Prader-Willi syndrome (PWS), will be featured in two poster presentations.

Key Points: 
  • Livoletide, a potential first-in-class treatment for Prader-Willi syndrome (PWS), will be featured in two poster presentations.
  • The company will also give an overview of its ongoing nevanimibe Phase 2b study in patients with classic congenital adrenal hyperplasia (CAH) in a third poster presentation.
  • Nevanimibe decreases adrenal steroidogenesis through the inhibition of ACAT1 and is being studied for the treatment of two orphan adrenal diseases: classic congenital adrenal hyperplasia (CAH) and endogenous Cushings syndrome (CS).
  • The company is currently advancing livoletide for the treatment of Prader-Willi syndrome and nevanimibe for the treatment of classic congenital adrenal hyperplasia and endogenous Cushings syndrome.

Millendo Therapeutics Announces ENDO 2019 Presentations on Livoletide for Prader-Willi Syndrome and Nevanimibe for Classic Congenital Adrenal Hyperplasia

Retrieved on: 
Thursday, March 14, 2019
Health, Biotechnology, Pharmaceutical, Adrenal gland disorders, Endocrine diseases, Organ systems, Endocrine system, Adrenal insufficiency, Congenital adrenal hyperplasia, Adrenal gland, Cushing's syndrome, CAH, Hyperplasia, Congenital adrenal hyperplasia due to 21-hydroxylase deficiency, Adrenal crisis

The company will also give an overview of its ongoing nevanimibe Phase 2b open-label study in patients with classic congenital adrenal hyperplasia (CAH) in a third session.

Key Points: 
  • The company will also give an overview of its ongoing nevanimibe Phase 2b open-label study in patients with classic congenital adrenal hyperplasia (CAH) in a third session.
  • For more information about Millendo Therapeutics pivotal study of livoletide (ZEPHYR) please visit www.clinicaltrials.gov ( NCT03790865 ).
  • Nevanimibe decreases adrenal steroidogenesis through the selective inhibition of ACAT1 and is being studied for the treatment of two orphan adrenal diseases: classic congenital adrenal hyperplasia (CAH) and endogenous Cushings syndrome (CS).
  • The company is currently advancing livoletide for the treatment of Prader-Willi syndrome and nevanimibe for the treatment of classic congenital adrenal hyperplasia and endogenous Cushings syndrome.